Shares of Praxis Precision Medicines (PRAX +13.25%) leaped on Monday after the clinical-stage biopharmaceutical company received a Breakthrough Therapy Designation (BTD) for its experimental tremor treatment.
By the close of trading, Praxis' stock price was up more than 13%.
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A promising development
Praxis specializes in using genetic insights to discover new therapies for neurological disorders. The biotech's efforts received a boost today when the U.S. Food and Drug Administration (FDA) granted a BTD for Praxis' ulixacaltamide treatment for patients with essential tremor.
The designation was granted based on the encouraging results from two recent phase 3 studies. The BTD, which is intended to accelerate the development of drugs that could substantially improve the treatment of serious medical conditions, should help to hasten a regulatory review of ulixacaltamide.
Praxis, in turn, plans to submit a New Drug Application for ulixacaltamide in early 2026.
"We recently completed a series of positive interactions with the FDA, that, together with this BTD, are enabling us to advance this promising treatment faster to patients," Praxis CEO Marcio Souza said in a press release.
NASDAQ: PRAX
Key Data Points
A significant unmet need
Approximately 7 million people are afflicted with essential tremor in the U.S. alone. The movement disorder typically leads to involuntary arm movements that can disrupt patients' lives. Current treatment options are not very effective and are often poorly tolerated.
"The granting of the Breakthrough Therapy Designation for ulixacaltamide ... further underscores its potential to address the substantial unmet need in patients with ET," Souza said.
